Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT06262113

A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1

The goal of this fully decentralized, randomized controlled trial is to compare the efficacy of two educational interventions for individuals with Neurofibromatosis 1 (NF1). The primary objective of the study is to determine which intervention leads to higher rates of evidenced-based health screenings for NF1 patients in primary care settings.

Adults with NF1 and parents/guardians of children with NF1 from across the U.S. who do not go to a specialized NF clinic and who have an upcoming annual wellness visits (e.g. an annual physical, a well-child visit, etc.) scheduled with a primary care provider (PCP) are eligible to enroll in the study. To see if you might be eligible, fill out a prescreening survey here: https://redcap.link/mynfguide

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Neurofibromatosis 1 Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Neoplasms Neoplasms by Histologic Type Neoplasms, Nerve Tissue Neoplastic Syndromes, Hereditary Nerve Sheath Neoplasms Nervous System Diseases Neurocutaneous Syndromes Neurodegenerative Diseases Neurofibroma Neurofibromatoses Neuromuscular Diseases Peripheral Nervous System Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

About this study

Background: The majority of individuals with Neurofibromatosis 1 (NF1) in the United States lack access to specialized NF1 clinics and consequently don't receive care aligned with national recommendations. To address this gap in care, researchers are evaluating two interventions to determine which one helps people get recommended NF1-related health screenings at their annual wellness visit with a primary care provider.

Methods: Participants (adult patients or parents) who enroll in the study will complete baseline survey assessments online before their PCP visit and then be randomized to one of two groups. Both groups will be given letters, one for themselves and one for their clinician, that describe NF1 care recommendations. After attending their annual wellness visit, all participants will be asked to complete an online follow-up survey. A small subsample of participants will also be asked to do a virtual qualitative interview. No visits to Massachusetts General Hospital are required for this study.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Adult Inclusion Criteria:

  • Currently lives in the United States (including Puerto Rico and other United States territories)
  • Has a clinical diagnosis of neurofibromatosis 1
  • Does not attend an NF clinic within the Children's Tumor Foundation NF Clinic Network
  • Has an in-person, well-person visit with a primary care provider scheduled within 3 months of their consent
  • Speaks English or Spanish

Parent/Guardian of a Child with NF1 Inclusion Criteria:

  • Currently lives in the United States (including Puerto Rico and other United States territories)
  • Cares for a child (age <18 years) with a clinical diagnosis of neurofibromatosis 1
  • Does not attend an NF clinic within the Children's Tumor Foundation NF Clinic Network
  • Their child has an in-person, well-person visit with a primary care provider scheduled within 3 months of their consent
  • Speaks English or Spanish

Exclusion criteria

  • Only one person per household may participate in the study
  • Unwilling or unable to give informed consent

Treatment and study plan

Letters about NF1 Care (Content Type 1)

Other

Participants will receive two letters about NF1 care, one for themselves and one for their primary care clinician, to read in advance of the patient's annual wellness visit

Letters about NF1 Care (Content Type 2)

Other

Participants will receive two letters about NF1 care, one for themselves and one for their primary care clinician, to read in advance of the patient's annual wellness visit

Primary outcomes

  1. Receipt of Recommended NF1 Health Screenings

    Time frame: 2 weeks after PCP visit

    Number of AAP and ACMG recommended health screenings received by the person with NF1 at the annual wellness visit with their PCP, as assessed by patient/parent self-report

Secondary outcomes

  1. Patient Activation Measure®

    Time frame: At baseline and 2 weeks after PCP visit

    The Patient Activation Measure will be used to assess patient's and parent's self-efficacy managing their or their child's care. The patient form has 13 items and the parent form has 10 items. The measure has scores ranging from 0-100, where higher scores indicate higher patient/parent activation (e.g. self-efficacy in managing your own or your child's healthcare).

  2. Consumer Assessment of Healthcare Providers and Systems (CAHPS®) Clinician and Group Survey Version 4.0 (Beta): Rating of the Visit

    Time frame: 2 weeks after PCP visit

    The CAHPS® Rating of the Visit will be used to assess patient/parent satisfaction with the visit. This rating is a single item scored from 0 to 10, where higher scores indicated a better visit.

  3. Consumer Assessment of Healthcare Providers and Systems (CAHPS®) Clinician and Group Survey Version 4.0 (Beta): How Well Providers Communicate With Patients Subscale

    Time frame: 2 weeks after PCP visit

    The subscale will be used to assess How Well Providers Communicate With Patients. This scale will be scored from 0 to 100, where higher scores indicated better communication with patients.

  4. Consumer Assessment of Healthcare Providers and Systems (CAHPS®) Clinician and Group Survey Version 4.0 (Beta): Providers' Use of Information to Coordinate Patient Care Subscale

    Time frame: 2 weeks after PCP visit

    The subscale will be used to assess Providers' Use of Information to Coordinate Patient Care. This scale will be scored from 0 to 100, where higher scores indicate better use of information to coordinate care.

Sponsors and collaborators

Lead sponsor

Massachusetts General Hospital

Other

Collaborators

  • Patient-Centered Outcomes Research Institute

Registry information

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Feb 15, 2024
Registry last updated
Jul 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.